​UK's First Huntington's Disease Clinical Trial Starts In Leeds

This week (Monday 15th July) Leeds Teaching Hospitals NHS Trust (LTHT) was the first site in the UK to have a patient enrolled into the global Roche Generation-HD1 Study. The study is investigating whether a new compound can be effective in the treatment of patients with Huntington's Disease (HD).

According to Huntington’s Disease Association (HDA) around one in 10,000 people have Huntington's.

Participants in the Phase 3 clinical trial, will undergo lumbar punctures every 2 months for 24 months, where a sample of brain fluid will be taken for analysis, after which, a trial drug (RG6042) or a placebo will be administered into the spinal fluid.

The trial drug is an ASO (antisense oligonucleotide). This is a short strand of genetic code that will bind to the mutant Huntington gene product (mHTT). It is hoped this process will lower the active mHTT levels in the brain and therefore, hopefully slow the progression of HD, and lead to a much-improved quality of life.

11 UK sites along with 15 countries are working towards recruiting 660 patients worldwide into the study, with 50 to 60 recruits coming from the UK.

Speaking this week, the UK’s first trialist in the study, 63 year old Paul Hammond said:

“It's really exciting to be involved in the study. I have known for around a year and a half that this treatment was on the way. My brother sent me the details of the study after seeing it on the news, so I spoke to my GP and he got me involved with the trial.

“My mum suffered from Huntington's Disease, and I found out in my 40’s that I was going to get sick. Now there is treatment on trial, it brings me hope, and it changes a lot of things for my family.

“I have two daughters in their 20’s, with one who has started a family of their own, and I feel now that I don't have to worry about their future due to this study.”

Co-ordinating the study at Leeds Teaching Hospitals NHS Trust, Callum Schofield, Genetics Research Practitioner, said:

“Generation-HD1 is the culmination of years of hard work for our HD team here in Leeds.

“It is a hugely ambitious inter-departmental effort, bringing together staff from across the Trust to work on what may be one of the definitive studies in the field of HD research.

“Obviously there is a long way to go yet, but we’re hopeful that this research can finally offer real, tangible hope to our HD families here in Yorkshire, and the HD community as a whole.

“Of course many people will not be able to take part in this research, as the scope for prospective participants is incredibly narrow, but it is important to note that this drug is being tested by other people on their behalf, indeed on behalf of the entire HD community.

“We are exceptionally grateful for our wonderful patients going into this trial, this wouldn’t be possible without them.”